Pacira BioSciences Announces PCRX-201 Granted Regenerative Medicine Advance Therapy (RMAT) Designation for the Treatment of Osteoarthritis of the Knee
Pacira BioSciences, Inc. receives RMAT designation from FDA for PCRX-201 gene therapy product candidate for osteoarthritis. Positive Phase 1 study results show efficacy for at least 52 weeks. Additional data to be presented at OARSI 2024. RMAT designation expedites development process.
Positive
PCRX-201 gene therapy product candidate receives RMAT designation from FDA for osteoarthritis treatment.
Phase 1 study with 72 patients shows efficacy of PCRX-201 for at least 52 weeks.
Co-administered steroid group demonstrates highest efficacy with improvements in pain and stiffness scores.
Preliminary data presented at OARSI 2023, 52-week data accepted for OARSI 2024.
RMAT designation accelerates development process with FDA guidance and potential for accelerated approval.
The RMAT designation for PCRX-201, a gene therapy candidate for osteoarthritis, by the FDA is an indication of the therapy's potential to address a significant unmet medical need in the treatment of this chronic condition. The therapy's mechanism of action, utilizing an intra-articular helper-dependent adenovirus to deliver interleukin-1 receptor antagonist, represents a novel approach in osteoarthritis management that could potentially modify the disease process rather than merely addressing symptoms.
From the perspective of medical research, the preliminary data suggesting sustained efficacy through 52 weeks is promising, especially considering the chronic nature of osteoarthritis and the limited long-term efficacy of current treatments. The reported improvements in WOMAC pain and stiffness scores, as well as KOOS functional assessment, are particularly relevant as these are established metrics for evaluating clinical outcomes in osteoarthritis trials. The co-administered steroid group's results may also provide insights into potential synergistic effects when combining gene therapy with existing treatment modalities.
The RMAT designation is likely to have a positive impact on Pacira BioSciences' market position and investor sentiment, as it not only highlights the therapeutic potential of PCRX-201 but also signals a smoother regulatory path forward. This could lead to increased investor confidence and potentially higher stock valuations for the company. The designation may also attract partnership opportunities and could enhance the company's bargaining power in potential licensing negotiations.
Furthermore, the osteoarthritis treatment market is substantial, with a growing prevalence due to the aging population and increasing obesity rates. A successful gene therapy could capture a significant market share, especially if it demonstrates a disease-modifying effect. The financial implications of such a therapy reaching the market could be considerable, with the potential for long-term revenue streams and a competitive edge in the non-opioid pain management sector.
Osteoarthritis is a common joint disorder, affecting millions globally and represents a significant burden on healthcare systems. The current treatment paradigm primarily focuses on symptom management with non-steroidal anti-inflammatory drugs (NSAIDs), analgesics and in more severe cases, joint replacement surgery. A gene therapy that can provide long-term relief and possibly alter the disease course could disrupt the market, shifting treatment strategies and potentially reducing the need for more invasive procedures.
Market research would focus on the adoption curve for new therapies, payer willingness to cover advanced treatments and the potential patient population that could benefit from PCRX-201. It is also important to consider the competitive landscape, where other biologics and novel therapeutics are in development. The RMAT designation may give Pacira a crucial time-to-market advantage, but the long-term market impact will depend on the final clinical trial results, pricing strategy and accessibility of the therapy.
03/13/2024 - 07:00 AM
-- PCRX-201 is the first gene therapy product candidate to receive RMAT designation for osteoarthritis --
-- Designation supported by encouraging preliminary data from 72-patient Phase 1 study --
-- 52-Week Data Accepted for Presentation at OARSI 2024 and 104-week data to be submitted for presentation later this year --
TAMPA, Fla., March 13, 2024 (GLOBE NEWSWIRE) -- Pacira BioSciences, Inc. (Nasdaq: PCRX), the industry leader in its commitment to non-opioid pain management and regenerative health solutions, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to PCRX-201 (enekinragene inzadenovec), the company’s novel, intra-articular helper-dependent adenovirus (HDAd) gene therapy product candidate that codes for interleukin-1 receptor antagonist (IL-1Ra), for the treatment of osteoarthritis of the knee.
“We are honored to receive FDA’s first-ever RMAT designation for a gene therapy product candidate in osteoarthritis," said Frank D. Lee, chief executive officer of Pacira BioSciences, Inc. “We continue to be encouraged by the preliminary clinical findings supporting PCRX-201 as a potential disease-modifying therapy for osteoarthritis, and we look forward to presenting additional follow-up data later this year.”
The company’s RMAT application was supported by the preliminary safety and efficacy findings from a Phase 1 open-label, proof-of-concept, single ascending dose trial that enrolled 72 patients in two three-dose cohorts: a co-administered intra-articular steroid cohort and a cohort that did not receive a steroid. PCRX-201 was well tolerated, with efficacy observed through at least 52 weeks at all doses and cohorts. The highest level of efficacy was achieved in the co-administered steroid group, which showed a greater percentage of patients with at least a 50% improvement in Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) pain and stiffness scores, as well as a meaningful improvement in (Knee Injury and Osteoarthritis Outcomes Score) KOOS functional assessment. Preliminary 36-week data were presented at the Osteoarthritis Research Society International (OARSI) 2023 World Congress, the premier annual international forum in osteoarthritis research and treatment. The 52-week data have been accepted for presentation at OARSI 2024 taking place in Vienna, Austria in April 2024 and the company expects to present 104-week efficacy and safety data later this year.
Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the development and review processes for promising therapies, including genetic therapies, that are intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and for which preliminary clinical evidence indicates that the drug or therapy has the potential to address an unmet medical need.
RMAT designation provides the benefits of intensive FDA guidance on efficient drug development, including the ability for early interactions with FDA to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the Biologics License Application (BLA), and other opportunities to expedite development and review. PCRX-201 was also granted Advanced Therapy Medicinal Products (ATMP) designation by the European Medicines Agency in May 2023.
About PCRX-201
PCRX-201 was acquired from GQ Bio Therapeutics GmbH, a privately held biopharmaceutical company headquartered in Hamburg, Germany. GQ Bio’s product candidates are next-generation gene transfer vehicles. These gene therapy vectors are highly efficient in entering joint cells to confer multi-year clinical benefit. In PCRX-201, the high-capacity adenoviral gene therapy vector codes for the expression of IL-1Ra, a cytokine inhibitor that plays a central role in blocking inflammation and catabolic processes that are associated with pain and disease progression in osteoarthritis. Its unique design includes an inducible promoter so that, only in the presence of inflammation signaling, the vector turns joint cells into factories to produce sustained therapeutic levels of IL-1Ra to manage pain and mitigate osteoarthritis-related joint damage while remaining localized to the joint space.
About Pacira
Pacira BioSciences, Inc. (Nasdaq: PCRX) is committed to providing a non-opioid option to as many patients as possible to redefine the role of opioids as rescue therapy only. Pacira has three commercial-stage non-opioid treatments: EXPAREL® (bupivacaine liposome injectable suspension), a long-acting local analgesic currently approved for infiltration, fascial plane block, and as an interscalene brachial plexus nerve block for postsurgical pain management; ZILRETTA® (triamcinolone acetonide extended-release injectable suspension), an extended-release, intra-articular injection indicated for the management of osteoarthritis knee pain; and ioveraº® , a novel, handheld device for delivering immediate, long-acting, drug-free pain control using precise, controlled doses of cold temperature to a targeted nerve. To learn more about Pacira, including the corporate mission to reduce overreliance on opioids, visit www.pacira.com .
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What is the RMAT designation granted to PCRX-201 by the FDA?
The FDA granted RMAT designation to PCRX-201, a gene therapy product candidate for osteoarthritis treatment.
How many patients were enrolled in the Phase 1 study for PCRX-201?
The Phase 1 study for PCRX-201 enrolled 72 patients.
What type of improvements were observed in the co-administered steroid group in the study?
The co-administered steroid group showed improvements in pain and stiffness scores, as well as functional assessment.
Where was the preliminary 36-week data presented?
The preliminary 36-week data for PCRX-201 were presented at the OARSI 2023 World Congress.
What benefits does RMAT designation provide for drug development?
RMAT designation provides benefits such as FDA guidance, potential for accelerated approval, and expedited development and review processes.
